AAV Production: IND-Enabling Documentation and Controls
Frequently Asked Questions
What level of plasmid DNA traceability is required for IND-enabling toxicology lots?
For materials supporting IND-enabling studies under GXP conditions, full traceability of the starting plasmid is expected. This includes documentation of the master cell bank origin, sequence verification of the plasmid, and a clear chain of custody from synthesis to transfection.
How are capsid and genome identity documented throughout the AAV production process?
Identity is confirmed at multiple stages. It begins with sequence verification of the input plasmids (e.g., rep/cap, ITR-genome). Post-production, analytical methods like next-generation sequencing (NGS) are used to confirm the integrity of the packaged genome, while mass spectrometry can verify capsid protein identity.
Applying proven intelligence in manufacturing controls is fundamental to accelerating the path for advanced therapeutics and complex biologics. For viral vector programs, particularly those involving AAV, establishing an unbroken chain of traceability from starting materials to the final drug product is a direct driver of regulatory success. This documentation provides the objective evidence that has supported our core scientific team’s 100% successful IND rate since 2019. This is a track record established by our scientific leadership prior to our 2024 launch, and it represents the standard of quality we continue at Franklin Biolabs.
The integrity of any AAV production run begins with the plasmid DNA. A verifiable and well-documented plasmid source is the foundation for a reproducible manufacturing process.
- Plasmid Sourcing: Full documentation of the master cell bank, sequence verification, and purity assessments.
- Lot-to-Lot Consistency: Controls to ensure the same plasmid lot is used for related production runs, or that any new lot is appropriately qualified.
- Chain of Custody: A complete record tracking the material from synthesis through to its use in cell culture.
In-process controls provide definitive data points that inform the viability of a batch and guide manufacturing decisions. Preclinical biodistribution studies can reveal complexities in vector behavior. Without complete traceability from the plasmid source to the final vialed material, interpreting these outcomes and justifying the safety profile to regulators becomes exceptionally difficult.
Final product characterization serves as the definitive record of what was produced. This analytical data package, generated within our specialized laboratory space, provides regulators with a comprehensive profile of the vector. It connects the specific batch used in IND-enabling toxicology studies to the material intended for clinical use, ensuring a clear and defensible link between preclinical safety data and the proposed human trial.
This rigorous approach to documentation and controls is a core component of our services. It provides the framework necessary to navigate regulatory expectations and move programs toward clinical evaluation with confidence.
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